- Topline results expected in 2Q 2025
Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical
company focused on advancing innovative treatments for spinal
muscular atrophy (SMA), cardiometabolic disorders, and other
serious diseases where protein growth factors play a fundamental
role, today announced it has completed enrollment in the Phase 2
EMBRAZE trial designed to show proof-of-concept of apitegromab, an
investigational selective myostatin inhibitor antibody, to safely
preserve lean muscle mass in individuals on GLP-1 receptor agonist
(GLP-1 RA) therapy for obesity. Results from this trial are
expected in the second quarter of 2025 and will inform the
development of SRK-439, a novel, investigational selective
myostatin inhibitor, which the Company plans to advance
specifically for the treatment of cardiometabolic disorders,
including obesity.
EMBRAZE is a randomized, double-blind, placebo-controlled, Phase
2 trial evaluating the efficacy, safety and pharmacokinetics of
apitegromab in adults who are overweight or obese without diabetes
and taking a GLP-1 RA. The trial fully enrolled participants aged
18-65 to assess the primary endpoint of change in lean mass by DEXA
scan at 24 weeks. Initiation of enrollment was announced in May
2024.
“We believe the rapid enrollment of the EMBRAZE trial speaks to
the unmet need to preserve lean muscle mass for patients
experiencing significant weight loss on GLP-1 therapies. As a
leader in myostatin inhibition, Scholar Rock is uniquely positioned
to play a meaningful role in the cardiometabolic space. Preserving
muscle has the potential to improve the quality of weight loss as
well as attenuate the weight regain often observed when people come
off GLP-1 therapy,” said Jing Marantz, M.D., Ph.D., Chief Medical
Officer at Scholar Rock. “We anticipate reporting results from
EMBRAZE in the second quarter of 2025 and plan to use the data to
further inform Scholar Rock’s development of SRK-439, which was
designed specifically for the treatment of obesity.”
The Company also announced today that it will present new
preclinical SRK-439 data in a poster presentation entitled,
“SRK-439 Selectively Inhibits Myostatin to Promote Healthy Body
Composition During Metformin Therapy” at The Obesity Society’s
Annual Meeting at ObesityWeek®, November 3-6, in San Antonio,
Texas. Preclinical data presented earlier showed that SRK-439
maintained lean mass and improved fat mass loss when used in
combination with a GLP-1 RA in diet-induced obesity (DIO) mice.
Preclinical data also supported the potential for SRK-439 to
increase lean mass and attenuate fat mass regain, thus contributing
to a favorable body composition following withdrawal from GLP-1 RA
treatment. The Company plans to file an IND for SRK-439 in
2025.
Apitegromab is being developed as potentially the first
muscle-targeted therapy for the treatment of spinal muscular
atrophy (SMA) and is the only muscle-targeted therapy to show
clinical proof-of-concept in SMA. The Company is on track to report
topline data in the fourth quarter of 2024 from the pivotal Phase 3
SAPPHIRE clinical trial in patients with SMA. If the trial is
successful and apitegromab is approved by the U.S. Food and Drug
Administration (FDA), the Company expects to initiate a commercial
product launch in 2025.
About Apitegromab
Apitegromab is an investigational fully human monoclonal
antibody inhibiting myostatin activation by selectively binding the
pro- and latent forms of myostatin in the skeletal muscle. It is
the first muscle-targeted treatment candidate to demonstrate
clinical proof-of-concept in spinal muscular atrophy (SMA).
Myostatin, a member of the TGFβ superfamily of growth factors, is
expressed primarily by skeletal muscle cells, and the absence of
its gene is associated with an increase in muscle mass and strength
in multiple animal species, including humans. Scholar Rock believes
that its highly selective targeting of pro- and latent forms of
myostatin with apitegromab may lead to a clinically meaningful
improvement in motor function in patients with SMA. The U.S. Food
and Drug Administration (FDA) has granted Fast Track, Orphan Drug
and Rare Pediatric Disease designations, and the European Medicines
Agency (EMA) has granted Priority Medicines (PRIME) and Orphan
Medicinal Product designations, to apitegromab for the treatment of
SMA. The efficacy and safety of apitegromab have not been
established and apitegromab has not been approved for any use by
the FDA or any other regulatory agency.
About the Phase 3 SAPPHIRE Trial in Spinal Muscular
Atrophy
SAPPHIRE is an ongoing randomized, double-blind,
placebo-controlled, Phase 3 clinical trial evaluating the safety
and efficacy of apitegromab in nonambulatory patients with Types 2
and 3 SMA who are receiving SMN-targeted therapy (either nusinersen
or risdiplam). SAPPHIRE targeted enrolling approximately 156
patients aged 2-12 years old in the main efficacy population. These
patients were randomized 1:1:1 to receive for 12 months either
apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by
intravenous (IV) infusion every 4 weeks. An exploratory population
that targeted enrolling up to 48 patients aged 13-21 years old will
also separately be evaluated. These patients were randomized 2:1 to
receive either apitegromab 20 mg/kg or placebo. For more
information about SAPPHIRE, visit www.clinicaltrials.gov.
Apitegromab has not been approved for any use by the US FDA or any
other health authority, and its safety and efficacy have not been
established.
About the Phase 2 EMBRAZE Trial in Obesity
EMBRAZE is a randomized, double-blind, placebo-controlled, Phase
2 proof-of-concept trial evaluating the efficacy, safety and
pharmacokinetics of apitegromab in adults with a body mass index
(BMI) of >27 (overweight) or a BMI of >30 (obese) and taking
a GLP-1 RA (tirzepatide or semaglutide). The target enrollment of
EMBRAZE is 100 subjects aged 18-65 who are overweight or obese
without diabetes. As part of the study design, the treatment period
is 24 weeks, and all subjects will receive a GLP-1 RA. In addition,
all subjects will be randomized 1:1 to receive either apitegromab
or placebo by intravenous (IV) infusion every four weeks during the
24-week treatment period. The primary endpoint is change from
baseline at Week 24 in lean mass assessed by dual-energy X-ray
absorptiometry. Secondary endpoints include additional weight loss
measures, safety and tolerability, and pharmacokinetic outcomes.
Exploratory endpoints at Weeks 24 and 32 include cardiometabolic
parameters (e.g. HbA1c), body composition, and physical
function.
About SRK-439
SRK-439 is a novel, preclinical, investigational myostatin
inhibitor that has high in vitro affinity for pro- and latent
myostatin and maintains myostatin specificity (i.e., no GDF11 or
Activin-A binding), and is initially being developed for the
treatment of cardiometabolic disorders, including obesity. Based on
preclinical data, SRK-439 has the potential to support healthier
weight management by preserving lean mass during weight loss. The
efficacy and safety of SRK-439 have not been established and
SRK-439 has not been approved for any use by the FDA or any other
regulatory agency.
About Scholar Rock
Scholar Rock is a biopharmaceutical company that discovers,
develops, and delivers life-changing therapies for people with
serious diseases that have high unmet need. As a global leader in
the biology of the transforming growth factor beta (TGFβ)
superfamily of cell proteins and named for the visual resemblance
of a scholar rock to protein structures, the clinical-stage company
is focused on advancing innovative treatments where protein growth
factors are fundamental. Over the past decade, Scholar Rock has
created a pipeline with the potential to advance the standard of
care for neuromuscular disease, cardiometabolic disorders, cancer,
and other conditions where growth factor-targeted drugs can play a
transformational role.
Scholar Rock is the only company to show clinical
proof-of-concept for a muscle-targeted treatment in spinal muscular
atrophy (SMA). This commitment to unlocking fundamentally different
therapeutic approaches is powered by broad application of a
proprietary platform, which has developed novel monoclonal
antibodies to modulate protein growth factors with extraordinary
selectivity. By harnessing cutting-edge science in disease spaces
that are historically under-addressed through traditional
therapies, Scholar Rock works every day to create new possibilities
for patients. Learn more about our approach at ScholarRock.com and
follow @ScholarRock and on LinkedIn.
Availability of Other Information About Scholar Rock
Investors and others should note that we communicate with our
investors and the public using our company website
www.scholarrock.com, including, but not limited to, company
disclosures, investor presentations and FAQs, Securities and
Exchange Commission filings, press releases, public conference call
transcripts and webcast transcripts, as well as on Twitter and
LinkedIn. The information that we post on our website or on Twitter
or LinkedIn could be deemed to be material information. As a
result, we encourage investors, the media and others interested to
review the information that we post there on a regular basis. The
contents of our website or social media shall not be deemed
incorporated by reference in any filing under the Securities Act of
1933, as amended.
Scholar Rock® is a registered trademark of Scholar Rock,
Inc.
Forward-Looking Statements
This press release contains "forward-looking statements" within
the meaning of the Private Securities Litigation Reform Act of
1995, including, but not limited to, statements regarding Scholar
Rock’s future expectations, plans and prospects, including without
limitation, Scholar Rock’s expectations regarding its growth,
strategy, progress and timing of its clinical trials for
apitegromab and its preclinical programs, including SRK-439, and
indication selection and development timing, including the
therapeutic potential, clinical benefits and safety thereof,
expectations regarding timing, success and data announcements of
current ongoing preclinical and clinical trials, expectations
regarding the achievement of important milestones, the ability of
any product candidate to perform in humans in a manner consistent
with earlier nonclinical, preclinical or clinical trial data, and
the potential of its product candidates and proprietary platform.
The use of words such as “may,” “might,” “could,” “will,” “should,”
“expect,” “plan,” “anticipate,” “believe,” “intend,” “future,”
“potential,” or “continue,” and other similar expressions are
intended to identify such forward-looking statements. All such
forward-looking statements are based on management's current
expectations of future events and are subject to a number of risks
and uncertainties that could cause actual results to differ
materially and adversely from those set forth in or implied by such
forward-looking statements. These risks and uncertainties include,
without limitation, that preclinical and clinical data, including
the results from the Phase 2 clinical trial of apitegromab are not
predictive of, may be inconsistent with, or more favorable than,
data generated from future or ongoing clinical trials of the same
product candidates, including, without limitation, the Phase 3
clinical trial of apitegromab in SMA; Scholar Rock’s ability to
provide the financial support, resources and expertise necessary to
identify and develop product candidates on the expected timeline;
the data generated from Scholar Rock’s nonclinical and preclinical
studies and clinical trials; information provided or decisions made
by regulatory authorities; competition from third parties that are
developing products for similar uses; Scholar Rock’s ability to
obtain, maintain and protect its intellectual property; Scholar
Rock’s dependence on third parties for development and manufacture
of product candidates including, without limitation, to supply any
clinical trials; and Scholar Rock’s ability to manage expenses and
to obtain additional funding when needed to support its business
activities and establish and maintain strategic business alliances
and new business initiatives, and our ability to continue as a
going concern; as well as those risks more fully discussed in the
section entitled "Risk Factors" in Scholar Rock’s Quarterly Report
on Form 10-Q for the quarter ended June 30, 2024, as well as
discussions of potential risks, uncertainties, and other important
factors in Scholar Rock’s subsequent filings with the Securities
and Exchange Commission. Any forward-looking statements represent
Scholar Rock’s views only as of today and should not be relied upon
as representing its views as of any subsequent date. All
information in this press release is as of the date of the release,
and Scholar Rock undertakes no duty to update this information
unless required by law.
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version on businesswire.com: https://www.businesswire.com/news/home/20240910921109/en/
Scholar Rock:
Investors Rushmie Nofsinger Scholar Rock
rnofsinger@scholarrock.com ir@scholarrock.com 857-259-5573
Media Molly MacLeod Scholar Rock mmacleod@scholarrock.com
media@scholarrock.com 802-579-5995
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